Phase I Study of Human Von Willebrand Factor for Von Willebrand's Disease
OBJECTIVES: I. Evaluate the effect of a new von Willebrand factor concentrate on bleeding time, in vivo recovery, and circulating half-life of the infused factor in patients with von Willebrand's disease.
II. Assess the safety of von Willebrand factor in these patients.
PROTOCOL OUTLINE: Patients receive 1 dose of von Willebrand factor concentrate. Timed blood studies are performed for the next 96 hours.
Patients are followed every 2 weeks for 16 weeks, and at 24, 36, and 52 weeks.
Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment
Von Willebrand's Disease
von Willebrand factor
Office of Rare Diseases (ORD)
Results (where available)
- Source: http://clinicaltrials.gov/show/NCT00004667
- Information obtained from ClinicalTrials.gov on July 15, 2010
Medical and Biotech [MESH] Definitions
Von Willebrand Disease, Type 1
A subtype of von Willebrand disease that results from a partial deficiency of VON WILLEBRAND FACTOR.
Von Willebrand Disease, Type 2
A subtype of von Willebrand disease that results from qualitative deficiencies of VON WILLEBRAND FACTOR. The subtype is divided into several variants with each variant having a distinctive pattern of PLATELET-interaction.
Von Willebrand Disease, Type 3
A subtype of von Willebrand disease that results from a total or near total deficiency of VON WILLEBRAND FACTOR.
Von Willebrand Factor
A high-molecular-weight plasma protein, produced by endothelial cells and megakaryocytes, that is part of the factor VIII/von Willebrand factor complex. The von Willebrand factor has receptors for collagen, platelets, and ristocetin activity as well as the immunologically distinct antigenic determinants. It functions in adhesion of platelets to collagen and hemostatic plug formation. The prolonged bleeding time in VON WILLEBRAND DISEASES is due to the deficiency of this factor.
Von Willebrand Diseases
Group of hemorrhagic disorders in which the VON WILLEBRAND FACTOR is either quantitatively or qualitatively abnormal. They are usually inherited as an autosomal dominant trait though rare kindreds are autosomal recessive. Symptoms vary depending on severity and disease type but may include prolonged bleeding time, deficiency of factor VIII, and impaired platelet adhesion.
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